JP2010540564A - 自己相補的なaavが媒介する眼の障害を処置または予防するための干渉rna分子の送達 - Google Patents

自己相補的なaavが媒介する眼の障害を処置または予防するための干渉rna分子の送達 Download PDF

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JP2010540564A
JP2010540564A JP2010527251A JP2010527251A JP2010540564A JP 2010540564 A JP2010540564 A JP 2010540564A JP 2010527251 A JP2010527251 A JP 2010527251A JP 2010527251 A JP2010527251 A JP 2010527251A JP 2010540564 A JP2010540564 A JP 2010540564A
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interfering rna
eye
scaav
vector
ocular
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JP2010540564A5 (enExample
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アラン アール. シェパード,
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アルコン リサーチ, リミテッド
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • A61P27/04Artificial tears; Irrigation solutions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • A61P27/06Antiglaucoma agents or miotics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • A61P27/14Decongestants or antiallergics
    • CCHEMISTRY; METALLURGY
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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Ophthalmology & Optometry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2010527251A 2007-10-01 2008-10-01 自己相補的なaavが媒介する眼の障害を処置または予防するための干渉rna分子の送達 Pending JP2010540564A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US97655207P 2007-10-01 2007-10-01
PCT/US2008/078380 WO2009046059A1 (en) 2007-10-01 2008-10-01 Self complementary aav-mediated delivery of interfering rna molecules to treat or prevent ocular disorders

Publications (2)

Publication Number Publication Date
JP2010540564A true JP2010540564A (ja) 2010-12-24
JP2010540564A5 JP2010540564A5 (enExample) 2012-09-13

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JP2010527251A Pending JP2010540564A (ja) 2007-10-01 2008-10-01 自己相補的なaavが媒介する眼の障害を処置または予防するための干渉rna分子の送達

Country Status (12)

Country Link
US (1) US20090087413A1 (enExample)
EP (1) EP2192926A1 (enExample)
JP (1) JP2010540564A (enExample)
KR (1) KR20100061792A (enExample)
CN (1) CN101815536A (enExample)
AU (1) AU2008308784B2 (enExample)
BR (1) BRPI0817937A2 (enExample)
CA (1) CA2694091A1 (enExample)
MX (1) MX2010001608A (enExample)
RU (1) RU2010117178A (enExample)
WO (1) WO2009046059A1 (enExample)
ZA (1) ZA201000423B (enExample)

Cited By (2)

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KR20170054493A (ko) * 2014-09-16 2017-05-17 젠자임 코포레이션 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터
JP2021526839A (ja) * 2018-06-12 2021-10-11 ザ ユニバーシティ オブ ブリストル 眼圧及び頭蓋内圧を調節するための材料及び方法

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EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
EP2287323A1 (en) * 2009-07-31 2011-02-23 Association Institut de Myologie Widespread gene delivery to the retina using systemic administration of AAV vectors
EA202091105A1 (ru) * 2010-04-23 2020-12-30 Юниверсити Оф Флорида Рисерч Фаундейшн, Инк. Композиции гуанилатциклазы и способы лечения врожденного амавроза лебера типа 1 (lca1)
CN103505743A (zh) * 2012-06-21 2014-01-15 北京命码生科科技有限公司 含功能性microRNA/siRNA的细胞微粒子及其应用
EP4012035B1 (en) * 2014-09-16 2024-11-06 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
CA2961523A1 (en) * 2014-09-16 2016-03-24 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
US20220403438A1 (en) 2019-10-08 2022-12-22 Trustees Of Boston College Proteins containing multiple, different unnatural amino acids and methods of making and using such proteins
MX2022010936A (es) 2020-03-05 2022-11-16 Neotx Therapeutics Ltd ³métodos y composiciones para el tratamiento del cáncer con células inmunológicas.
EP4161583A1 (en) * 2020-06-05 2023-04-12 Institut National de la Santé et de la Recherche Médicale (INSERM) Methods and pharmaceutical compositions for treating ocular diseases

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US20040209832A1 (en) * 2001-11-30 2004-10-21 Mcswiggen James RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20040198682A1 (en) * 2001-11-30 2004-10-07 Mcswiggen James RNA interference mediated inhibition of placental growth factor gene expression using short interfering nucleic acid (siNA)
US20050048529A1 (en) * 2002-02-20 2005-03-03 Sirna Therapeutics, Inc. RNA interference mediated inhibition of intercellular adhesion molecule (ICAM) gene expression using short interfering nucleic acid (siNA)
WO2005019422A2 (en) * 2003-08-13 2005-03-03 The Board Of Trustees Of The University Of Illinois Silencing of tgf-beta receptor type ii expression by sirna
US7947267B2 (en) * 2004-10-08 2011-05-24 Potentia Pharmaceuticals, Inc. Viral complement control proteins for eye disorders
TWI386225B (zh) * 2004-12-23 2013-02-21 Alcon Inc 用於治療眼睛病症的結締組織生長因子(CTGF)RNA干擾(RNAi)抑制技術
TWI401316B (zh) * 2004-12-23 2013-07-11 Alcon Inc 用於治療青光眼之血清澱粉樣蛋白A的RNAi抑制作用
TW200639253A (en) * 2005-02-01 2006-11-16 Alcon Inc RNAi-mediated inhibition of ocular targets
BRPI0609206A2 (pt) * 2005-03-11 2010-03-02 Alcon Inc inibiÇço mediada por rnai de proteÍna relacionada frizzled-1 para tratamento de glaucoma
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JPN6013023942; 'Ocular gene transfer with self-complementary AAV vectors.' Invest Ophthalmol Vis Sci. 48(7), 2007, 3324-3328 *
JPN6013023944; 'Mechanisms of AAV transduction in glaucoma-associated human trabecular meshwork cells.' J Gene Med. 8(5), 2006, 589-602 *
JPN6013023945; 'AAV hybrid serotypes: improved vectors for gene delivery.' Curr Gene Ther. 5(3), 2005, 299-310 *
JPN6013023948; 'Inhibition of Ocular Angiogenesis by siRNA Targeting Vascular Endothelial Growth Factor Pathway Gene' Am J Pathol. 165(6), 2004, 2177-2185 *
JPN6013023949; 'Potential applications for RNAi to probe pathogenesis and develop new treatments for ocular disorder' Gene Therapy 13(6), 2006, 559-562 *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20170054493A (ko) * 2014-09-16 2017-05-17 젠자임 코포레이션 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터
JP2017529395A (ja) * 2014-09-16 2017-10-05 ジェンザイム・コーポレーション ミオシリン(myoc)緑内障を処置するためのアデノ随伴ウイルスベクター
KR102612871B1 (ko) 2014-09-16 2023-12-13 젠자임 코포레이션 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터
JP2021526839A (ja) * 2018-06-12 2021-10-11 ザ ユニバーシティ オブ ブリストル 眼圧及び頭蓋内圧を調節するための材料及び方法
JP7430917B2 (ja) 2018-06-12 2024-02-14 ザ ユニバーシティ オブ ブリストル 眼圧及び頭蓋内圧を調節するための材料及び方法

Also Published As

Publication number Publication date
CA2694091A1 (en) 2009-04-09
AU2008308784B2 (en) 2013-07-18
ZA201000423B (en) 2011-03-30
BRPI0817937A2 (pt) 2015-04-07
MX2010001608A (es) 2010-03-15
US20090087413A1 (en) 2009-04-02
WO2009046059A1 (en) 2009-04-09
KR20100061792A (ko) 2010-06-09
RU2010117178A (ru) 2011-11-10
AU2008308784A1 (en) 2009-04-09
CN101815536A (zh) 2010-08-25
EP2192926A1 (en) 2010-06-09

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